A Clinical Study of ANX005 and IVIG in Subjects With Guillain Barré Syndrome (GBS)

A Phase 1b Study to Evaluate the Safety, Tolerability and Drug-Drug Interactions of ANX005 and Intravenous Immunoglobulin (IVIg) in Subjects With Guillain Barré Syndrome

This study is a multi center, open-label, study of ANX005 in combination with IVIg in subjects diagnosed with GBS.

Study Overview

• Status: Completed
• Conditions: Guillain-Barré Syndrome
• Intervention / Treatment: Drug: ANX005; Drug: Intravenous immunoglobulin

Detailed Description

The study will enroll approximately 12 subjects into one cohort, with subjects receiving 75 mg/kg of ANX005, a dose shown to be safe and well-tolerated as monotherapy and that provides a meaningful level of exposure to be tested in combination with IVIg. Dosing of IVIg will be administered as is the current standard of care (0.4 g/kg/day x 5 days) and ANX005 will be administered concurrently, beginning on Day 1 or 2 of the treatment period. Subjects will be followed for 6 months after treatment for observation and evaluation.

• Study Type: Interventional
• Enrollment (Actual): 14
• Phase: Phase 1

Contacts and Locations

Study Locations

• Bangladesh
  • Dhaka, Bangladesh
    • National Institute of Neurosciences and Hospital
• Denmark
  • Aarhus, Denmark
    • Aarhus University Hospital

Participation Criteria

Eligibility Criteria

• Ages Eligible for Study: 16 years and older (Adult, Older Adult)
• Accepts Healthy Volunteers: No
• Genders Eligible for Study: All

Description

Inclusion Criteria:

• Diagnosis of GBS according to the National Institute of Neurological Disorders and Stroke Diagnostic Criteria for Guillain Barré Syndrome
• Onset of GBS-related weakness ≤14 days prior to infusion
• GBS-DS score of 3, 4, or 5

Exclusion Criteria:

• Clinically significant findings that may interfere with the conduct of the study or the interpretation of the data
• Be at risk of suicide or self-harm
• Received previous treatment with plasma exchange for GBS
• Any diagnosis of a variant of GBS
• Have a history of anaphylaxis or severe systemic response to immunoglobulin
• Documented, clinically significant, pre-existing polyneuropathy from another cause
• Clinically significant intercurrent illness, medical condition, or medical history
• History of chronic use of steroid or immunosuppressant medication
• Active alcohol, drug, or substance abuse
• Females who are pregnant, breast feeding, or unable or unwilling to use highly effective methods of contraception throughout the study

Study Plan

How is the study designed?

Design Details

• Primary Purpose: Treatment
• Allocation: N/A
• Interventional Model: Single Group Assignment
• Masking: None (Open Label)

Number of Arms

1

Arms and Interventions

Participant Group / Arm	Intervention / Treatment
Experimental: Open Label Treatment Arm; One (1) dose of ANX005, 75 mg/kg, will be administered IV. IVIg, 0.4 g/kg, will be administered for 5 consecutive Days.	Drug: ANX005; investigational drug; Other Names: Anti C1q Antibody; Drug: Intravenous immunoglobulin; investigational drug; Other Names: IVIg

What is the study measuring?

Primary Outcome Measures

Outcome Measure	Measure Description	Time Frame
Safety and tolerability of ANX005 when administered in combination with IVIg: incidence of TEAEs, SAEs, AE's	As measured by incidence of TEAEs, SAEs, AE's related to ANX005, SAE's related to ANX005, Grade 3 or higher AEs, Grade 3 or higher AEs related to ANX005, AEs leading to study or treatment discontinuation.	6 months

Secondary Outcome Measures

Outcome Measure	Measure Description	Time Frame
Pharmacokinetics of ANX005 when administered in combination with IVIg	As measured by ANX005 serum concentrations	3 months
Pharmacodynamics of ANX005 when administered in combination with IVIg	As measured by CH50 and C1q serum concentrations	4 months
Values and change from baseline in Guillain-Barré Syndrome Disability Score (GBS-DS) by visit	The 6-point Guillain-Barré Syndrme Disability Score (GBS-DS) is a widely accepted and easily obtainable scoring system used to assess functional status of GBS subjects. The score is as follows: 0 = Healthy, 1 = Minor symptoms and capable of running, 2 = Able to walk independently 10 meters or more but unable to run, 3=Able to walk more than 10 meters across an open space with help, 4 = Bedridden or chair bound, 5 = Needing mechanical ventilation, 6 = death	6 months

Collaborators and Investigators

• Sponsor: Annexon, Inc.
• Collaborators: International Centre for Diarrhoeal Disease Research, Bangladesh; ResearchPoint Global
• Investigators: Study Director: Henk-André Kroon, MD, MBA, Annexon, Inc.

Study record dates

Study Major Dates

• Study Start (Actual): January 29, 2020
• Primary Completion (Actual): May 19, 2021
• Study Completion (Actual): May 19, 2021

Study Registration Dates

• First Submitted: July 11, 2019
• First Submitted That Met QC Criteria: July 26, 2019
• First Posted (Actual): July 29, 2019

Study Record Updates

• Last Update Posted (Actual): August 11, 2021
• Last Update Submitted That Met QC Criteria: August 9, 2021
• Last Verified: August 1, 2021

More Information

Terms related to this study

• Additional Relevant MeSH Terms: Pathologic Processes; Nervous System Diseases; Immune System Diseases; Autoimmune Diseases of the Nervous System; Demyelinating Diseases; Autoimmune Diseases; Disease; Neuromuscular Diseases; Peripheral Nervous System Diseases; Polyradiculoneuropathy; Polyneuropathies; Syndrome; Guillain-Barre Syndrome; Physiological Effects of Drugs; Immunologic Factors; Antibodies; Immunoglobulins; Immunoglobulins, Intravenous; gamma-Globulins; Rho(D) Immune Globulin
• Other Study ID Numbers: ANX005-GBS-03

Plan for Individual participant data (IPD)

• Plan to Share Individual Participant Data (IPD)?: No

Drug and device information, study documents

• Studies a U.S. FDA-regulated drug product: Yes
• Studies a U.S. FDA-regulated device product: No